Genetic cure
Gene therapy would help children get rid of Hurler's syndrome, an unusual disease that normally leads to death before age 10. Such patients lack an enzyme that clears out the molecular waste in cells. The increased waste results in mental retardation and visible deformities. A team of researchers led by Mike Dexter at Christie Hospital in Manchester has started a gene therapy course on three children. They aim to provide the children with a gene that secretes the missing enzyme called alpha- l- iduronidase . The researchers are expected to insert good copies of the gene into bone marrow cells of the patients. They aim is to introduce the gene into stem cells that mature into white blood cells. Dexter expects that the method would help cure the patients for good because stem cells last throughout adult life and renew themselves as time passes. They suggest that the treatment should be started before patients show any physical sign of disease ( New Scientist , Vol 155, No 2093).